Novartis and Amgen axe BACE drug for Alzheimer's

Another Alzheimer’s disease drug has been axed after Novartis, Amgen and the Banner Alzheimer’s Institute decided to end two pivotal studies involving a medicine targeting BACE.

The organisations have ended development of the BACE1 (beta secretase 1) inhibitor CNP520 – or umibecestat – which was in two phase 2/3 studies in the Alzheimer's Prevention Initiative Generation Program.

They made the decision after assessment of unblinded data during a regular pre-planned review identified worsening in some measures of cognitive function.

CNP520 was being tested for safety and efficacy in the prevention or delay of the onset of Alzheimer's in people at high risk of developing symptoms based on their age and genetic status.

Study sponsors are informing investigators of the decision to discontinue the clinical program of CNP520 in Alzheimer's prevention, and advising that participants should stop taking the investigational treatment.

The clinical investigators will contact participants to discuss what happens next, including follow-up appointments as appropriate.

It’s the latest in a long list of Alzheimer’s drug failures, with no new medicines making it to market since 2003 when Forest’s Namenda was approved by the FDA.

Pharma has been trying to find ways to stop or slow the disease from spreading by targeting the amyloid plaques that are commonly seen in the brains of people with Alzheimer’s.

However this latest failure adds to the growing body of evidence that targeting amyloid may not be the solution – BACE inhibitors work by preventing creation of a precursor protein to amyloid.

Big pharma is also turning its back on BACE along with the first generation drugs directly targeting the rogue protein itself – AstraZeneca and Eli Lilly axed development of their BACE drug lanabecestat.

Results from CNP520 are in line with other BACE drugs, which have been associated with slight worsening of symptoms.

Pharma’s strategy is now to refine its approach to targeting amyloid, such as AZ and Lilly’s MEDI1814 targeting the most toxic form of the protein, or focusing on another approach together such as the “tau” tangles also associated with the disease.